SAN DIEGO – Bleeding episodes and need for treatment were more frequent among boys with types 1 and 2 von Willebrand disease.
The U.S. hemophilia drug market, which serves about 20,000 patients, is worth $4.6 billion a year.
Emicizumab is now approved in Europe and the United States for routine prophylaxis of bleeding episodes in people with factor VIII inhibitors.
“There aren’t a lot of options available to Medicaid programs in terms of controlling costs, because we don’t set the initial costs”
The FDA has granted breakthrough therapy designation to a gene therapy product aimed at hemophilia A.
The drug was approved by the U.S. Food and Drug Administration in November 2017.
Dr. Cynthia Dunbar, of the NIH, talks about the “critical mass of successes” in hematology and what is coming in the future.
From the Journals
The phase 2 trial included patients who did not respond to erythropoiesis-stimulating agents.
Eltrombopag, from Novartis, will get expedited review for first-line treatment of severe aplastic anemia. It is already approved as a second-line...
ATLANTA – Successfully treating Waldenström macroglobulinemia often resolves acquired von Willebrand disease.